骨髓纤维化（MF）是一种慢性骨髓增生性肿瘤，通常表现为逐渐恶化的衰弱症状，对患者的生活质量产生负面影响。由于其严重性、持续性和普遍性，疲劳是一种多因素且繁重的 MF 相关症状，其中贫血是一个促成因素和主要未满足的需求。 Janus 激酶 (JAK)1/JAK2/激活素 A 受体 1 型抑制剂 momelotinib 的临床试验显示，除了改善 MF 相关症状外，还具有一致的贫血益处。针对有症状和贫血患者的 3 期 MOMENTUM 试验达到了主要终点，与达那唑相比，莫莫替尼在第 24 周时骨髓纤维化症状评估表 (MFSAF) 总症状评分 (TSS) 降低 ≥50% 的比例更高。为了支持积极的主要终点结果，我们对 MOMENTUM 患者报告的结果进行了纵向、应答者和事件发生时间分析，根据 MFSAF、欧洲癌症研究和治疗组织生活质量问卷 (EORTC) 进行测量QLQ-C30）和患者报告结果测量信息系统（PROMIS）评估。这些分析表明，莫莫替尼具有快速、持久的反应优势，在第 29 天时实现了首次 TSS 反应，并随着时间的推移持续改善。对于每个 MFSAF 单个项目，莫莫洛替尼与达那唑的改善更有利，并且在第 24 周观察到与疾病和癌症相关的疲劳和身体机能有更大的改善，在 3 个评估中的多个项目/领域取得了显着结果。这些研究结果一致证明，莫莫替尼可提供显着的症状益处。作者版权所有 © 2023。由 Wolters Kluwer Health, Inc. 代表欧洲血液学协会出版。

Myelofibrosis (MF) is a chronic myeloproliferative neoplasm that typically manifests with debilitating symptoms that progressively worsen, negatively impacting patients' quality of life. Fatigue is a multifactorial and burdensome MF-related symptom due to its severity, persistence, and prevalence, with anemia a contributing factor and major unmet need. Clinical trials of the Janus kinase (JAK)1/JAK2/activin A receptor type 1 inhibitor momelotinib have shown consistent anemia benefits, in addition to improvements in MF-related symptoms. The phase 3 MOMENTUM trial in symptomatic and anemic patients met its primary end point, with a greater proportion having a Myelofibrosis Symptom Assessment Form (MFSAF) Total Symptom Score (TSS) reduction ≥50% at week 24 with momelotinib versus danazol. To support the positive primary end point result, we conducted longitudinal, responder, and time-to-event analyses of patient-reported outcomes from MOMENTUM, as measured by the MFSAF, European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30), and Patient-Reported Outcomes Measurement Information System (PROMIS) assessments. These analyses demonstrated rapid and durable response benefits with momelotinib, with achievement of first TSS response by day 29 and continued improvement over time. Improvements favored momelotinib versus danazol for each MFSAF individual item, and greater improvements were observed for disease- and cancer-related fatigue and physical functioning at week 24, with significant results for multiple items/domains across the 3 assessments. These findings are consistent in demonstrating that momelotinib provides substantial symptom benefit.Copyright © 2023 the Author(s). Published by Wolters Kluwer Health, Inc. on behalf of the European Hematology Association.